Major therapeutic advances have been made in patients with episodic and progressive
cerebellar ataxias, downbeat nystagmus and some vestibular disorders. We provide an update review on this subject highlighting important research findings from the last two years.
RECENT FINDINGS: Recently, the use of
omaveloxolone for 2 years significantly improved upright stability in
Friedreich's ataxia patients. In an open-label study,
N-acetyl-l-leucine administered for 6-weeks significantly improved clinical impression of change,
ataxia, and quality of life in patients with
Niemann-Pick disease type C1. A 12-week treatment with
dalfampridine was associated with improved standing balance in a subgroup of patients with
multiple sclerosis. A
gluten-free diet alone improved
ataxia in half of patients with antiglutamic
acid decarboxylase (GAD)
ataxia, suggesting that
gluten sensitivity might be part of the underlying pathogenesis in anti-GAD
ataxia. In a head-to-head trial, both prolonged-release
4-aminopyridine (4-AP) and
acetazolamide effectively reduced the attacks up to 60% in patients with
episodic ataxia type 2 (EA2), albeit 4-AP had fewer adverse effects. Small observational studies have shown that patients with episodic vestibular syndrome who cannot be diagnosed as definite or probable vestibular
migraine, might still improve vestibular symptoms following preventive treatment for
migraine. The use of
vitamin D supplementation in
benign paroxysmal positional vertigo,
steroids in acute unilateral vestibulopathy, and
betahistine in
Ménière's disease patients remains controversial.
SUMMARY: Although the use of several
therapies is being established in the treatment of cerebellar and vestibular disorders, there is an urgent need for prospective controlled therapeutic trials.